Moving Closer to an Approved Therapy for NTM Lung Disease: FDA Advisory Committee says Arikayce (Inhaled Liposomal Amikacin) is a safe and effective treatment for those with limited options

Posted on August 08, 2018   |   
Like 6 Likes

As you know currently there are no FDA-approved treatments specifically for nontuberculous mycobacterial (NTM) lung disease caused by MAC. Yesterday, the Antimicrobial Drugs Advisory Committee of the U.S. Food & Drug Administration met to assess a newly proposed treatment, Arikayce (Inhaled Liposomal Amikacin) for NTM lung disease caused by Mycobacterium avium complex (MAC).

During the meeting, the Advisory Committee heard presentations from Insmed, the company who developed the treatment, the FDA staff who reviews and analyzes data from the treatment’s clinical trials and from the public. Remarkably, 12 community members traveled to the FDA to tell their stories, each having been allotted 5 minutes during the open public hearing session. The physicians, patients, family members and representatives from the COPD Foundation and our partner, NTMir, spoke of the urgent need for new treatment options that are more effective and less toxic. They explained how NTM has changed their life and what it meant to them to have a better chance at achieving culture conversion, the main benefit of the proposed new treatment.

Their voices, along with the dozens of patients who submitted written comments prior to the meeting, were heard loud and clear and we want to say THANK YOU!

The Advisory Committee was asked to vote on three questions including whether the primary outcome of culture conversion was clinically meaningful, whether the data showed that Arikayce was safe and effective for all NTM lung disease caused by MAC, and whether Arikayce was safe and effective for NTM lung disease cause by MAC in those who have not achieved culture conversion on standard antibiotic regimens. There was a great deal of discussion about what it meant to a patient to have a negative culture and how that translates to feeling better and doing better. In the end, the Committee voted 8 to 6 in support of culture conversion being a meaningful benefit.

Most importantly, the Committee voted 12 to 2 that Insmed had shown that Arikayce was a safe and effective treatment for those who have limited or no other treatment options. Since all of the patients who participated in the trials had already been on standard therapy, the Committee did not vote favorably that it was safe and effective for all patients but further studies that include newly diagnosed or untreated patients could change this in the future.

We are very pleased with the positive outcome that reflects the strong data that Insmed presented for those patients with nowhere else to turn. While this is not the final say in the approval of the therapy, the final decision will be made by the FDA’s Division of Anti-Infective Products, the COPD Foundation is optimistic that they will follow suit after reviewing the data, results, and comments by the research and the patient community.

The COPD Foundation, along with NTMir has worked tirelessly to bring about change in this realm and we are grateful to Insmed for their dedication to finding solutions that can help this community. We believe an approval of the first drug specifically for NTM lung disease will catalyze additional interest in the area and spur more innovation that can bring benefits to even greater shares of the affected community. We are so appreciative to the FDA and Insmed for their effectiveness of moving this forward and listening to the patient voice and perspective.

The FDA will issue their final decision on or before September 28th. Stay tuned for more updates and more discussion about the FDA Meeting, the review processes they use and how YOUR voice has played a role in the development and review of this important treatment. If you would like to read more about the recent FDA Advisory Committee meeting, please click here for the FDA’s meeting materials. Feel free to ask questions here on BronchandNTM360social and a member of our team will do our best to answer!